Actively Recruiting
A Phase I/IIa, Single Site, Open-Label, Ascending Dose Study to Evaluate the Safety and Efficacy of OPF-310 in Adults With Type 1 Diabetes Mellitus
Led by Otsuka Pharmaceutical Factory, Inc. · Updated on 2026-03-02
13
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating OPF-310, which consists of encapsulated porcine islet cells for xenotransplantation, in adults with unstable Type 1 Diabetes Mellitus (T1DM). This first-in-human study aims to assess the safety, tolerability, and effectiveness of OPF-310 transplantation and to determine the recommended Phase 2 dose (RP2D). Eligible participants have experienced severe hypoglycemic episodes despite treatment with a closed loop system (CLS) for at least six months. Participants will receive OPF-310 in an open-label trial with ascending doses. Initially, three subjects will receive 6,000 islet equivalents (IEQ)/kg and three will receive 12,000 IEQ/kg, both followed by safety monitoring. Then, seven more subjects will receive the recommended Phase 2 dose determined from earlier results. A total of 13 patients will be transplanted with OPF-310. During the study, researchers will monitor participants for up to one year after transplant. They will assess outcomes such as reduction in severe hypoglycemic events, improvements in glucose control measured by continuous glucose monitoring (CGM), insulin use, and HbA1c levels. Other evaluations include porcine C-peptide levels, psychological impact assessments, and hypoglycemia awareness. Safety and tolerability will also be closely observed throughout the study period.
CONDITIONS
Brief Title
A Study to Investigate Safety and Effectiveness of Porcine Pancreatic Cells (OPF-310) in Patients With Type 1 Diabetes Mellitus
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 35 to 65 years inclusive at the time of signing informed consent
- Established diagnosis of Type 1 Diabetes Mellitus for at least 5 years according to American Diabetes Association criteria
- Unstable Type 1 Diabetes Mellitus not controlled after receiving a closed loop system (specific CGM and insulin pumps) for at least 6 months
- History of severe (Level 3) hypoglycemic episodes three or more times in the past year, or one episode with Clarke Score 64, or TBR >1% and Clarke Score 64
- C-peptide less than 0.3 ng/mL following a mixed meal tolerance test or undetectable fasting C-peptide
- Hemoglobin A1C (HbA1c) of 9.0% or lower
- Use of contraception consistent with local regulations for clinical study participation
- Willingness to cooperate with lifetime follow-up after transplantation
- Ability to provide signed informed consent
You will not qualify if you...
- History of insulin resistance with average insulin dose requirement 3 0.8 unit/kg/day for 1 week prior to enrollment
- Diagnosis of latent autoimmune diabetes in adults (LADA), ketosis-prone diabetes, or maturity onset diabetes of the young (MODY)
- C-reactive protein (CRP) level 3 10 mg/L
- Clinically unstable thyroid disease or abnormal thyroid stimulating hormone (TSH) levels unresolved by treatment
- History of malignancies in past 5 years except basal or squamous cell carcinoma
- Positive tests for HIV, hepatitis B, or hepatitis C
- Active or untreated proliferative diabetic retinopathy unless treated successfully
- Serious conditions affecting study participation including recent peripheral vascular disease amputation, heart failure NYHA class II-IV, chronic obstructive pulmonary disease or asthma requiring hospitalization, macroalbuminuria, or low kidney function (eGFR < 30 ml/min)
- Use of warfarin or anticoagulants except aspirin, or abnormal blood clotting tests
- Adrenal insufficiency requiring corticosteroids
- Previous pan-peritonitis
- Previous cardiovascular or cerebrovascular disease including recent heart attack or coronary artery disease
- Hematopoietic stem cell abnormalities
- Recent blood transfusions or anticipated surgery requiring transfusion
- Previous organ, skin, or tissue transplant from human or animal donor
- Treatment with immunosuppressive medication
- Abdominal surgery within 12 weeks prior to enrollment except uncomplicated procedures
- Use of hypoglycemic medications other than insulin
- Treatment with acetaminophen or hydroxycarbamide
- Use of investigational products within 12 weeks or 5 half-lives prior to enrollment
- Allergy to antibiotics used in OPF-310 manufacturing or allergies to insulin, pork, or alginate/seaweed
- Panel reactive antibodies (PRA) > 80%
- Active drug, substance, or alcohol addiction
- Body mass index (BMI) above 27 kg/m2
- Any condition that may interfere with study adherence including dementia, psychiatric disorder, or history of non-adherence
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 year after transplant
Participants receive a transplant of OPF-310 porcine pancreatic cells at doses determined by the study phase, followed by safety monitoring.
Multiple visits during the first year after transplant as part of safety and efficacy monitoring
Duration - Up to 1 year after transplant
Participants are monitored for long-term safety and effectiveness, including assessments of blood sugar control and psychological impact.
Regular visits for up to 52 weeks post-transplant
Trial Site Locations
Total: 1 location
1
University of Illinois Hospital & Health Sciences System
Chicago, Illinois, United States, 60612
Actively Recruiting
Research Team
F
For participant-focused inquiries, please contact:
F
For physician or professional inquiries, please contact:
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here